How do genetically engineered drugs help people with hemophilia?

Answer 1

The production of clotting factor proteins (the treatment for hemophilia) has many different parts. While much of the clotting factor protein used to treat hemophilia is recombinant (I'll explain this term in the next paragraph) I feel it important to also point out that a lot of the medications are still plasma derived, basically just separated off from human blood.

The recombinant protein molecules that are used to treat hemophilia (for the sake of the question we'll use the term drug, even though it would more accurately be called a biologic) are created in a lab, not being pulled out of human blood. The molecules themselves are not exactly being genetically engineered either. To produce these protein molecules, specific cells have been genetically engineered to create them and excrete them as a biproduct. These cells are cultured in large vats where the protein molecules are collected.

Advantages to these "recombinant" medications are in the purity and perceived safety. Since the protein molecules are not derived from blood the chance of contamination from a contaminated blood supply can be eliminated. The ability to culture these cells and produce the proteins in this manner also prevent the supply of the medication from relying on the blood supply, which throughout recent history has varied greatly as natural catastrophes, wars, and many other situations may deplete the blood supply.

Answer 2

Doctors can replace the people with hemophilia by replacing the defective on the X chromosome. Then the blood wouldn't clot normally.